General Updates | Investigating a menin inhibitor in pediatric acute leukemia: a sub-trial of the PedAL trial

This is a study we’re very excited about. The Leukemia and Lymphoma Society sponsored the initial research for this molecule when it was in the laboratory in Michigan. And Dr Grembecka’s work then led to our sponsoring a company through our therapy acceleration program that became Cura Oncology. Then Cura worked within our Beat AML program to start studies in adult AML. And now, while those studies are still ongoing, we are starting the pediatric development. So this is, from start to finish, the power of what we’ve been able to do. And it is the way we’d like to see development happen, where, you know, as soon as you know the dosage and safety in adults, that you start thinking about how to do pediatrics. 

And the PedAL trial now, again, it’s a single trial, the same trial, a single database, European Medicines Agency and the FDA agreeing to the same trial. And we’ve already, we opened it in, you know, early in the spring and we’re already five patients have been treated. So in this rare subset, this is really exciting. This is just a Phase Ib, but we’re already making plans for the Phase II. And again, this is the kinds of trials that are for registrational intent so that we can get this drug available to children as soon as possible. 

We believe that because pediatric leukemias are rare, while you can test a half a dozen menin inhibitors in adults, you really can’t test all of them effectively in the pediatric population. And so we are testing this one and there will be, you know, an understanding of how it compares to other menin inhibitors in the pediatric setting. And that will include what the toxicities are, what the formulation is, what the dose levels are, how hard it is to give for kids. And so I think these are really important considerations that this trial will happen. And then the only other thing I’d say is that we have some really amazing pediatric resources for patients and for families at LLS, and providers should feel free to have their patients and their patient families reach out to us for support, for financial assistance, for information, and for access to trials. We’re happy to help.

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